
02/09/2025
We may have a cure for diabetes.
A patient with type 1 diabetes is now producing his own insulin after receiving a groundbreaking transplant of gene-edited pancreatic cells, without needing any anti-rejection drugs.
This is the first time in humans that donor islet cells have been genetically modified to evade the immune system entirely. Type 1 diabetes is caused when a person’s immune system mistakenly attacks the islet cells in their pancreas, which are responsible for insulin production.
Treatments typically involve lifelong insulin injections and, in rare cases, transplants, though these usually require immunosuppressants, which carry serious long-term risks. In this new case, a 42-year-old man who had lived with type 1 diabetes since childhood received injections of donor islet cells into his forearm. But before that, scientists made three specific edits using CRISPR: two removed cellular markers that help T cells recognize foreign tissue, and one added a protein called CD47, which helps block the body's innate immune responses.
The results were dramatic. Over 12 weeks, the edited cells began producing insulin in response to glucose spikes, like after meals. The man didn’t need any immunosuppressive medication to keep the cells alive. Not all the edited cells survived, only those with all three edits remained functional, which gave researchers a built-in comparison and proof that their editing approach worked. While the patient didn’t receive enough cells to fully manage his condition without additional treatment, the experiment shows that functional, gene-edited cell transplants can survive and work in the human body without being attacked by the immune system.
paper
"Survival of Transplanted Allogeneic Beta Cells with No Immunosuppression" New England Journal of Med. (2025)